A 13-year-old Belgian boy, Lucas Jemeljanova, is reported cured of diffuse intrinsic pontine glioma (DIPG) after his tumour vanished following treatment in the French BIOMEDE clinical trial. DIPG is a uniformly lethal brainstem cancer in children - median survival nine to ten months and under 10% alive at two years - where surgery is impossible. BIOMEDE, launched in 2014, compared erlotinib, everolimus and dasatinib and uniquely required a needle molecular biopsy for each patient to guide therapy. Lucas was randomized to everolimus, an mTOR inhibitor, and his MRI scans showed progressive disappearance of the tumour; of 233 trial patients seven outlived expectations but only Lucas became radiologically tumour-free. He later stopped the drug on his own and the cancer has not returned.
Researchers at Gustave Roussy led by Dr. Jacques Grill are investigating why Lucas responded so completely, hypothesizing a very rare mutation that made his tumour dependent on the mTOR pathway. Teams are generating tumour organoids that replicate his cancer’s genetics to test that dependency. BIOMEDE 2.0, begun in September 2022, now focuses on everolimus, and separate immunotherapy trials have produced notable shrinkage with at least one other child tumour-free for over four years. The case underscores the power of biopsy-driven, molecularly stratified trial design to expose rare, curative responses hidden in aggregate studies.
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